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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03786783




Registration number
NCT03786783
Ethics application status
Date submitted
24/12/2018
Date registered
26/12/2018

Titles & IDs
Public title
Dinutuximab, Sargramostim, and Combination Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma
Scientific title
A Pilot Induction Regimen Incorporating Chimeric 14.18 Antibody (ch14.18, Dinutuximab) (NSC# 764038) and Sargramostim (GM-CSF) for the Treatment of Newly Diagnosed High-Risk Neuroblastoma
Secondary ID [1] 0 0
NCI-2018-03732
Secondary ID [2] 0 0
NCI-2018-03732
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Ganglioneuroblastoma 0 0
High Risk Neuroblastoma 0 0
Condition category
Condition code
Cancer 0 0 0 0
Neuroendocrine tumour (NET)
Cancer 0 0 0 0
Children's - Other
Cancer 0 0 0 0
Children's - Brain
Cancer 0 0 0 0
Brain

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Surgery - Autologous Hematopoietic Stem Cell Transplantation
Treatment: Drugs - Carboplatin
Treatment: Drugs - Cisplatin
Treatment: Drugs - Cyclophosphamide
Treatment: Drugs - Dexrazoxane
Treatment: Other - Dinutuximab
Treatment: Drugs - Doxorubicin
Treatment: Drugs - Etoposide
Treatment: Other - External Beam Radiation Therapy
Treatment: Drugs - Isotretinoin
Treatment: Drugs - Melphalan
Treatment: Other - Sargramostim
Treatment: Drugs - Thiotepa
Treatment: Drugs - Topotecan
Treatment: Drugs - Vincristine

Experimental: Treatment(chemotherapy, dinutuximab, sargramostim, ASCT, EBRT) - See Detailed Description


Treatment: Surgery: Autologous Hematopoietic Stem Cell Transplantation
Undergo ASCT

Treatment: Drugs: Carboplatin
Given IV

Treatment: Drugs: Cisplatin
Given IV

Treatment: Drugs: Cyclophosphamide
Given IV

Treatment: Drugs: Dexrazoxane
Given IV

Treatment: Other: Dinutuximab
Given IV

Treatment: Drugs: Doxorubicin
Given IV

Treatment: Drugs: Etoposide
Given IV

Treatment: Other: External Beam Radiation Therapy
Undergo EBRT

Treatment: Drugs: Isotretinoin
Given PO

Treatment: Drugs: Melphalan
Given IV

Treatment: Other: Sargramostim
Given SC

Treatment: Drugs: Thiotepa
Given IV

Treatment: Drugs: Topotecan
Given IV

Treatment: Drugs: Vincristine
Given IV
Intervention code [1] 0 0
Treatment: Surgery
Intervention code [2] 0 0
Treatment: Drugs
Intervention code [3] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants With Unacceptable Toxicity
Timepoint [1] 0 0
Up to the first 5 cycles of treatment
Primary outcome [2] 0 0
Percentage of Participants Who Are Feasibility "Failure"
Timepoint [2] 0 0
Up to the first 5 cycles of treatment
Secondary outcome [1] 0 0
Response Rate
Timepoint [1] 0 0
Up to the first 5 cycles of treatment
Secondary outcome [2] 0 0
Event-free Survival
Timepoint [2] 0 0
Up to 1 year
Secondary outcome [3] 0 0
Overall Survival
Timepoint [3] 0 0
Up to 1 year

Eligibility
Key inclusion criteria
* Patients must be enrolled on ANBL00B1 or APEC14B1 prior to enrollment on ANBL17P1.
* Patients must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites. The following disease groups are eligible:

* Patients with International Neuroblastoma Risk Group (INRG) stage M disease are eligible if found to have either of the following features:

* MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features; OR
* Age > 547 days regardless of biologic features;
* Patients with INRG stage MS disease with MYCN amplification
* Patients with INRG stage L2 disease with MYCN amplification
* Patients > 547 days of age initially diagnosed with INRG stage L1, L2 or MS disease who progress to stage M without prior chemotherapy may enroll within 4 weeks of progression to stage M.
* Patients >= 365 days of age initially diagnosed with MYCN amplified INRG stage L1 disease who progress to stage M without systemic therapy may enroll within 4 weeks of progression to stage M.
* Patients initially recognized to have high-risk disease must have had no prior systemic therapy (other than topotecan/cyclophosphamide initiated on an emergent basis and within allowed timing as described).
* Patients observed or treated with a single cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (e.g., as per ANBL0531, ANBL1232 or similar) for what initially appeared to be non-high risk disease but subsequently found to meet the criteria will also be eligible.
* Patients who receive localized emergency radiation to sites of life-threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis will be eligible.
* Creatinine clearance (CrCl) or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/sex as follows:

* Age 1 month to < 6 months (male 0.4 mg/dL, female 0.4 mg/dL)
* Age 6 months to < 1 year (male 0.5 mg/dL, female 0.5 mg/dL)
* Age 1 to < 2 years (male 0.6 mg/dL, female 0.6 mg/dL)
* Age 2 to < 6 years (male 0.8 mg/dL, female 0.8 mg/dL)
* Age 6 to < 10 years (male 1 mg/dL, female 1 mg/dL)
* Age 10 to < 13 years (male 1.2 mg/dL, female 1.2 mg/dL)
* Age 13 to < 16 years (male 1.5 mg/dL, female 1.4 mg/dL)
* Age >= 16 years (male 1.7 mg/dL, female 1.4 mg/dL) (within 7 days prior to enrollment).
* Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment).
* Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 10 x ULN. For the purposes of this study, ULN for ALT is 45 IU/L (within 7 days prior to enrollment).
* Shortening fraction of >= 27% by echocardiogram (within 7 days prior to enrollment).
* Ejection fraction of >= 50% by echocardiogram or radionuclide angiogram (within 7 days prior to enrollment).
* No known contraindication to peripheral blood stem cell (PBSC) collection. Examples of contraindications might be a weight or size less than the collecting institution finds feasible, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure.
* All patients and/or their parents or legal guardians must sign a written informed consent.
* All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met.
Minimum age
No limit
Maximum age
30 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Patients >18 months of age with INRG stage L2, MYCN non-amplified, regardless of additional biologic features.
* Patients with bone marrow failure syndromes.
* Patients that are >= 12 and =< 18 months of age with INRG stage M and all 3 favorable biologic features (i.e., non-amplified MYCN, favorable pathology, and deoxyribonucleic acid [DNA] index > 1) are not eligible.
* Patients on immunosuppressive medications (e.g. tacrolimus, cyclosporine, corticosteroids for reasons other than prevention/treatment of allergic reactions, adrenal replacement therapy, etc.) are not eligible.
* Female patients who are pregnant are ineligible since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential.
* Lactating females who plan to breastfeed their infants.
* Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method during study therapy and for two months after the last dose of ch14.18 (dinutuximab) are not eligible.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
4/03/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
22/09/2025
Actual
Sample size
Target
Accrual to date
Final
42
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 0 0
Royal Children's Hospital - Parkville
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
District of Columbia
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
New York
Country [5] 0 0
United States of America
State/province [5] 0 0
Pennsylvania
Country [6] 0 0
United States of America
State/province [6] 0 0
Tennessee
Country [7] 0 0
United States of America
State/province [7] 0 0
Utah
Country [8] 0 0
New Zealand
State/province [8] 0 0
Auckland

Funding & Sponsors
Primary sponsor type
Government body
Name
National Cancer Institute (NCI)
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Sara M Federico
Address 0 0
Children's Oncology Group
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://grants.nih.gov/policy/sharing.htm


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03786783